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December 8, 2025BloodOpen Access

Venetoclax and azacytidine in childhood primary advanced myelodysplastic syndromes, refractory/relapsed Acute Myeloid Leukemia and therapy-related myeloid diseases

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Authors

GPGennaro PaganoEMEdoardo MuratoreFVFrancesca Vendemini

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Overview

Retrospective analysis finds venetoclax with azacytidine achieves 58% complete response in childhood advanced myeloid diseases, implying promising outcomes for future protocols.

Key Points

  • This study aimed to evaluate the efficacy of venetoclax combined with azacytidine in pediatric advanced myeloid malignancies.
  • Retrospective multicenter study conducted on 50 childhood patients with myeloid diseases.
  • Patients received venetoclax and azacytidine treatment with continuous assessment of response via bone marrow evaluations.
  • Adverse events were recorded according to CTCAE v5.0 grading.
  • 58% of patients achieved a complete response after treatment.
  • Adverse events were reported in 34% of patients, without any treatment-related deaths.
  • The study highlighted significant event-free survival rates, especially for patients bridged to transplantation.

Cite This Study

Pagano et al. (2025) studied this question.

synapsesocial.com/papers/69362f484fa91c937236d700https://doi.org/10.1182/blood-2025-289
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Clinical activity of venetoclax and azacitidine in children with de novo or secondary multiple relapsed/refractory acute myeloid leukemia: a real-world experience2025 · 2 citations
  2. 2Venetoclax in combination with cytarabine with or without idarubicin in children with relapsed or refractory acute myeloid leukemia: A multi-center phase II expansion study2025
  3. 3Phase II study of clinical efficacy of venetoclax in combination with azacitidine in patients with therapy related myelodysplastic syndrome (t-MDS)2025
  4. 4Clinical outcomes of hypomethylating agents and venetoclax in newly diagnosed unfit and relapsed/refractory paediatric, adolescent and young adult acute myeloid leukaemia patients2024 · 10 citations
  5. 5A phase I/II trial of azacitidine and venetoclax in previously untreated Myelodysplastic Syndromes2025 · 1 citations